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An Extension Study to Evaluate Casimersen or Golodirsen in Patients With Duchenne Muscular Dystrophy
study id #: NCT03532542
condition: Duchenne Muscular Dystrophy
status: enrolling by invitationpurpose:
The main objective of this study is to evaluate the safety and tolerability of long-term treatment with casimersen or golodirsen in patients with Duchenne muscular dystrophy (DMD).
intervention: Casimersen, Golodirsen
mechanism of action: Exon-skipping to promote dystrophin production
last updated: February 26, 2019
start date: August 2, 2018
estimated completion: June 30, 2026
phase of development: Phase 3
size / enrollment: 260
- Number of patients with serious adverse events (SAEs) [Time Frame: Up to 30 days after the last infusion of study drug (assessed up to 148 weeks)]]
• Completed a clinical trial evaluating casimersen or golodirsen, per protocol.
• Is between 7 and 23 years of age, inclusive, at enrollment.
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